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Novel RNA-targeted therapies for hereditary ATTR amyloidosis and their impact on the autonomic nervous system
PURPOSE: Transthyretin-mediated hereditary amyloidosis (hATTR amyloidosis) is a multisystemic disease with heterogeneous clinical presentation. Hallmarks of the disease are sensory-motor and autonomic neuropathy and cardiomyopathy. Two disease-modifying drugs, inotersen (an antisense oligonucleotide...
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Springer Berlin Heidelberg
2019
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6763402/ https://www.ncbi.nlm.nih.gov/pubmed/31399774 http://dx.doi.org/10.1007/s10286-019-00626-8 |